Painful Lessons
Authors: Fintan Steele
Journal: Molecular Therapy (2000)

Gene therapy is getting a lot of press lately, much to the chagrin of its practitioners. If there was anyone left in the field who still believed gene therapy would be easy, the recent tragic events at the University of Pennsylvania have decisively put an end that fantasy. Perhaps that partially explains the rush to judgment, both private and very public, in response to the still mysterious death of Jesse Gelsinger. This premature judgment is unfair at best and destructive at worst. First, we must dismiss the question of guilt or innocence for now, i.e., did the UPenn researchers err? Despite early “evidence” provided almost entirely through the Washington Post, New York Times, and Philadelphia Inquirer, we don't know. The facts are not out. There is a process underway to uncover the facts and arrive at a conclusion. There will be a resolution, but it is certainly made more difficult by the cacophony of congressional hearings, FDA leaks, skewed newspaper articles, and rampant rumors. We must step back, reserve judgment, and let the process run its course rather than impeding its progress. Nevertheless, we can derive two early lessons from this tragedy. The first and most obvious is that rather than worrying about the effects of the UPenn incident on the field of gene therapy, each researcher needs to look closely at his or her own program and make sure it is “squeaky clean.” Granted, the current onerous regulatory procedures need reform and streamlining, but given the intense public scrutiny of gene therapy, it is important that gene therapists adhere tightly to the regulations in place now. The bottom line is that we are, in essence, developing drugs, and the admittedly imperfect regulatory procedures already in place have worked in the past. Careful attention to procedural details will also help illuminate—and maybe eliminate—the murky regulatory area that lies between the RAC and the FDA. Even more importantly, careful reporting will assist other researchers in the design and interpretation of their ongoing trials and may prevent further incidents such as occurred at UPenn. The second lesson we need to take to heart is perhaps not as obvious, but is just as critical. The uneven, maybe god-awful, press coverage underlines the fact that we are not doing enough to educate the general public about what we do. Granted, this is very difficult in the face of basic public misinformation about genetics: Someone who won't think twice about swallowing an experimental pill recoils at the thought of changing his or her genetic makeup. Unfortunately, this is more our problem than anyone else's. Granted, researchers have been misquoted in the past and this will inevitably happen again. Rather than retreat, we must engage the public and the press even more aggressively—make them understand what we do and why, as well as all the uncertainties that haunt us daily. There will be other adverse events in future gene therapy trials: Helping the public understand the science behind the risks will make them less politically charged, although certainly no less tragic for the pioneers involved.